Fulcrum Therapeutics, Inc. (NASDAQ:FULC) Q3 2023 Earnings Call Transcript

Operator: Our next question comes from the line of Dae Gon Ha with Stifel. Your line is open.

Benazir Ali: Hi, this is Benazir Ali for Dae Gon. Thanks for taking our question. Just a couple on pociredir. How challenging has it been to resign some of the prior trial sites to participate in the trial at this point? Can you share some color on that? And what are some of the questions that they’re having about, the clinical hold and how they can feel more comfortable about drug safety? And then a follow up to that is we know that you’re not exactly sure about when you’re going to have data on pociredir, but is it more likely to be mid-year or yearend or somewhere in between, if not at a conference, maybe just a presentation through the company? Thank you.

Alex Sapir: Yeah, that’s great. Thanks, Benazir and let me start and then Iain, please feel free to jump in if I miss anything. So, yeah, so just as a quick reminder, we had seven sites in the Phase 1B study prior to the initiation of the hold and just as a reminder, that was an all-comer study. So our plan specific, and they were all U.S. sites, our plan specifically in the U.S. is to double the number of sites specifically in the U.S., as well as to look outside of the U.S. as well and we feel that we need to go to a greater number of sites simply because we have a more narrowly defined inclusion-exclusion criteria. So it will be a more difficult trial to enrol than we have in the past. I think what we’ve been hearing from physicians, and I’d like Iain to comment on this as well, but I think what we’ve been hearing from physicians is the benefits that this drug, as an oral HBF inducer, can provide to patients, right.

We’ve been able to show up until now that with six weeks of dosing at a 12-milligram dose, we can get patients to total fetal hemoglobin levels of 25 and I think that’s really exciting for the sites and the physicians that are going to participate in this study, and I think it’s also very exciting for the patients as well. I think in terms of when we think we’ll be able to show that, I think right now our focus has really been on engaging with the centers. Once we have those centers stood up, we’ve got a couple of IRBs that have already approved it. We have one site that is ready to start receiving drug, and we’re also doing a lot with the community, sickle cell community, in and around those sites to really sort of educate the community about this very, very interesting study that the center is now participating in.

So that’s really been our focus. I think at this point, Benazir, I think if I was to give you sort of any indication, the only thing I would know with great certainty is that that indication or that guidance would be incorrect. So I think give us a couple of months. Let us get a couple of months of patient enrolment under our belt, as I’ve said in the past, and I think at that point we’ll be in a much better position to provide more specific guidance that I feel more comfortable about. So anything else you think around the centers, Iain?

Iain Fraser: Yeah, thanks, Alex. I can provide a little bit of color around the types of sites themselves and as Alex alluded to earlier, it was an all-comer study, and so we were at a number of sites that started out essentially as pediatric sites because of the way that sickle cell disease care is administered, a lot of the pediatric sites hold on to their older patients as they go into adulthood because they don’t have great partners to transfer those patients to and so we had a number of those sites within the study originally. Those are obviously somewhat younger patients overall, less treatment experience, and less severe impact of disease, just given the duration that they’ve experienced. And what we’re finding as we go out is that a number of those sites feel that they don’t have quite the level of severity or previous treatment experience that we might need for the protocol and so those are sites that are not continuing, but some of them clearly still do have those patients.